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Launch delays hamper near-term impact of GPS experimentation program

Launch delays hamper near-term impact of GPS experimentation program

Yahoo21-03-2025
The Space Force is looking for ways to experiment with new technologies on its next-generation GPS satellites, but persistent delays to a key demonstration program could limit its options.
The service planned to launch the Navigation Technology Satellite-3 demonstration, dubbed NTS-3, in 2022 with an eye toward experimenting with new positioning, navigation and timing signals and payloads that could be installed on future GPS satellites and shape its long-term plans for the constellation.
The satellite's development, led by the Air Force Research Lab and L3Harris, has proceeded on schedule, but delays to the rocket assigned to fly the spacecraft — United Launch Alliance's new Vulcan Centaur — have stalled the program for years. The mission is slated to fly on Vulcan's first national security launch this year, but those plans are on hold as the company awaits final certification from the Space Force.
Cordell DeLaPena, who oversees the Space Systems Center's positioning, navigation and timing and satellite communications portfolios, said the service is weighing its options for how to proceed with integrating NTS-3 technology into upcoming GPS production lines.
'The longer it takes to actually launch those experiments, get the data and be able to assess it, the window starts to close on the availability of production vehicles,' he told Defense News in an interview.
The Space Force had intended to funnel NTS-3-proven capabilities into the production line for its latest variant of GPS satellites, dubbed GPS IIIF. The service plans to buy 20 of these satellites from Lockheed Martin and, to date, has ordered 10. The first five of those spacecraft are slated for deliveries over a five-year period beginning in 2027.
ULA's new rocket won't fly its first Space Force missions until 2025
DeLaPena said GPS IIIF is approaching the end of its design period and will soon shift toward production. There's still room on the satellite for additional size, weight and power — or SWAP — which means the program could still make changes to incorporate NTS-3 technology. But the clock is ticking, he said.
'If there are a handful of these experiments that launch and prove themselves out on orbit and if they're mature enough to start considering maturing those concepts for production, that would be the path,' DeLaPena said.
If the the Space Force misses its window to install NTS-3 technology on the first five GPS IIIF satellites, the service could aim to include any relevant technology either on its next batch of five spacecraft or as part of other PNT programs, DeLaPena said. That includes a program called Resilient GPS, which is meant to augment the larger constellation with a fleet of small, lightweight, lower-cost satellites.
The Space Force's NTS-3 demonstration and its plans for Resilient GPS, or R-GPS, are part of a broader rethinking of its approach to providing navigation and timing capabilities. One piece of that involves the orbit in which satellites reside.
The military has traditionally launched its GPS satellites to medium Earth orbit, and that's where R-GPS will operate. However, the service is considering a multi-orbit approach for its future PNT capabilities. Along those lines, NTS-3 is destined for geosynchronous orbit, and the Space Development Agency plans to launch PNT satellites to low Earth orbit as part of its Proliferated Warfighter Space Architecture.
DeLaPena noted that demonstrating a 'blended,' multi-orbit navigation capability is a primary goal for NTS-3, adding that countries like Japan, South Korea and India are all exploring GEO-based systems.
The Space Force is in the midst of an analysis of alternatives that will further define a roadmap for the service's future mix of PNT capabilities. The need for an R-GPS capability was an outgrowth of that study, which should be completed this summer, DeLaPena said.
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Alnylam to Present Progress in Transforming the Treatment of Cardiovascular Disease with RNAi Therapeutics at European Society of Cardiology Congress 2025
Alnylam to Present Progress in Transforming the Treatment of Cardiovascular Disease with RNAi Therapeutics at European Society of Cardiology Congress 2025

Business Wire

time14 hours ago

  • Business Wire

Alnylam to Present Progress in Transforming the Treatment of Cardiovascular Disease with RNAi Therapeutics at European Society of Cardiology Congress 2025

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present new data from its hypertension and transthyretin amyloidosis (ATTR) programs at the upcoming European Society of Cardiology (ESC) Congress 2025, taking place in Madrid, Spain, from August 29 - September 1, 2025. These presentations will highlight the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and reinforce Alnylam's commitment to advancing innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options. Highlights include data from Cohort A of the KARDIA-3 Phase 2 study evaluating the investigational RNAi therapeutic, zilebesiran, in patients with uncontrolled hypertension and high cardiovascular risk. This analysis will be presented as a late-breaking abstract in the Hot Line 4 session on August 30, 2025. Data from the Company's flagship TTR franchise will also be presented, including new analyses from the HELIOS-B Phase 3 study of AMVUTTRA ® (vutrisiran), which delivers rapid knockdown of transthyretin, in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). These presentations include results of 12-month follow-up from the ongoing open-label extension (OLE) period of HELIOS-B, which will provide further insights into the sustained longer-term benefits of treatment with AMVUTTRA, and an analysis examining the effect of AMVUTTRA on days lost to death and/or hospitalization (DLDH). ESC Congress Presentation Details Hypertension Abstracts KARDIA-3: Zilebesiran as add-on therapy in adults with hypertension and established cardiovascular disease or at high cardiovascular risk Hot Line 4 Session Presentation Time: Saturday, August 30, 2025, 4:30 – 4:45 pm (CEST), 10:30 – 10:45 am (EDT) Presenting Author: Neha Pagidipati, U.S. Room: Madrid, Main Auditorium Impact of zilebesiran, an investigational RNA interference therapeutic targeting hepatic angiotensinogen, on renin–angiotensin system biomarkers in patients with mild-to-moderate hypertension Moderated ePoster: Treatment of Hypertension: Randomized Controlled Trials Time: Saturday, August 30, 2025, 5:15 – 6:00 pm (CEST), 11:15 am – 12:00 pm (EDT) Presenting Author: Michael A Weber, U.S. Room: Station 4, Research Gateway ATTR Abstracts Vutrisiran reduces days lost to death and/or hospitalization versus placebo in patients with transthyretin amyloidosis with cardiomyopathy in the HELIOS-B trial Moderated ePoster: Multidisciplinary Care in Heart Failure Time: Sunday, August 31, 2025, 9:15 – 10:00 am (CEST), 3:15 – 4:00 am (EDT) Presenting Author: Mazen Hanna, U.S. Room: Station 3, Research Gateway HELIOS-B: 12-month results from the open-label extension period of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy Abstract Session: Therapeutic Advances in Amyloid Cardiomyopathy Time: Sunday, August 31, 2025, 11:15 – 11:25 am (CEST), 5:15 – 5:25 am (EDT) Presenting Author: Pablo Garcia-Pavia, Spain Room: Science Box 2 (Research Gateway) Investor Webcast Information Alnylam management will discuss the KARDIA-3 results in a live event which will be webcast on August 30, 2025, at 1:00 p.m. ET (7:00 pm CEST). The webcast will be available on the Investors section of the Company's website at An archived webcast will be available on the Alnylam website approximately two hours after the event. AMVUTTRA ® (vutrisiran) INDICATION AND IMPORTANT SAFETY INFORMATION Indications In the EU, AMVUTTRA ® (vutrisiran) is indicated for the treatment of: hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase. About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. It is marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and it is also approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in the US, Europe, Brazil, Japan and UAE. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR). For US Media only: For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit For EU Media: For the full EU Summary of Product Characteristics, see About Zilebesiran Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of hypertension to reduce cardiovascular risk in high unmet need populations. Zilebesiran targets angiotensinogen (AGT), the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a role in blood pressure (BP) regulation. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein, and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables infrequent biannual subcutaneous dosing, increased selectivity and the potential to achieve continuous control of BP to impact cardiovascular risk. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche. About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide. 1-4 About Cardiovascular Disease and Hypertension Cardiovascular disease (CVD) is a global health crisis and a leading cause of death worldwide, responsible for approximately 20 million deaths annually. 5,6 Hypertension is the primary cause of and number one modifiable risk factor for CVD. 7 An estimated 1 in 3 adults worldwide have hypertension, and, despite wide availability of antihypertensives, up to 80% of all patients, and up to a third of treated patients, do not reach and maintain blood pressure (BP) targets. 8 Even when blood pressure appears well managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage. 9-15 These patients require novel approaches that not only reduce BP, but also lower overall cardiovascular risk. About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. 16 Its discovery has been heralded as 'a major scientific breakthrough that happens once every decade or so,' and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. 17 By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. 16 This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its ' Alnylam P 5 x25 ' strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Alnylam Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam's expectations regarding the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and genetic and other conditions; Alnylam's ability to advance innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options; the safety and efficacy of vutrisiran for the treatment of ATTR-CM and the safety and efficacy of zilebesiran for the treatment of hypertension; and Alnylam's ability to achieve its ' Alnylam P 5 x25 ' strategy should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam's ability to successfully execute on its ' Alnylam P 5 x25 ' goals; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the 'Risk Factors' filed with Alnylam's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam's subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638. 2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112. 3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9. 4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. 5 GBD 2021 Causes of Death Collaborators. Lancet. 2024;403:2100-2132. 6 Lindstrom M, DeCleene N, Dorsey H, et al. J Am Coll Cardiol. 2022;80:2372-2425. 7 Yusuf S, Joseph P, Rangarajan S, et al. Lancet. 2020;395:795-808. 8 NCD Risk Factor Collaboration (NCD-RisC). Lancet. 2021;398:957-980. 9 Ebinger JE, Driver M, Ouyang D, et al. eClinicalMedicine. 2022;48:101442. 10 Kario K. Prog Cardiovasc Dis. 2016;59:262-281. 11 Doumas M, Tsioufis C, Fletcher R, et al. J Am Heart Assoc. 2017;6:e006093. 12 Mezue K, Goyal A, Pressman GS, et al. J Clin Hypertens. 2018;20:1247-1252. 13 Rothwell PM, Howard SC, Dolan E, et al. Lancet. 2010;375:895-905. 14 Tatasciore A, Renda G, Zimarino M, et al. Hypertension. 2007;50:325-332. 15 Mokadem ME, Boshra H, El Hady YA, et al. J Hum Hypertens. 2019;34:641-647. 16 Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498. 17 Zamore P. Cell. 2006;127(5):1083-1086.

H-1Bs are wreaking havoc on American workers
H-1Bs are wreaking havoc on American workers

The Hill

time14 hours ago

  • The Hill

H-1Bs are wreaking havoc on American workers

When it comes to immigration, there's a refrain that periodically arises with respect to new immigrants: 'They're even more American than us,' or something to that effect. And if immigration causes any ill effects on Americans already here — such as disruptions in the economy or employment environment — they are reminded that they should just grit their teeth and 'learn to code.' Unfortunately, that advice may no longer be helpful. Layoffs in the tech industry for 2025 had already exceeded 80,000 as of July, according to estimates. Although the public may know the tech climate has been bleak, they haven't heard much about the causes. Corporate executives have been eager to insinuate that AI is driving the employment environment. Amazon CEO Andy Jassy said in June that there would 'be fewer people doing some of the jobs that the technology actually starts to automate.' However, Jassy did not mention another factor — the employment data indicate that Amazon has led corporate America in spurning U.S. workers in favor of foreign-born alternatives. The company's main operating arm submitted 31,817 Labor Condition Applications for H-1B, H-1B1, and E-3 visas in the second quarter of fiscal 2025, according to data published by the Labor Department's Office of Foreign Labor Certification. The number grows higher — to 40,757 — if one accounts for Amazon Web Services, the company's cloud-management division. NVIDIA placed a distant second with 27,244 applications. Other top offenders included Goldman Sachs, which used three entities to file more than 26,000 applications. There's also Microsoft (14,181 applications) and Apple (8,393 applications). Labor Condition Applications allow companies to fill roles based in the U.S. with foreign laborers. They recently came under fire from Vice President JD Vance. Alluding to Microsoft's announcement of layoffs weeks earlier, Vance said at an event, 'You see some Big Tech companies where they'll lay off 9,000 workers, and then they'll apply for a bunch of overseas visas.' He added that he 'just found out' and had 'not yet had that conversation with Microsoft.' In a statement, Microsoft denied that Americans had been laid off as a result of their foreign alternatives, pointing to the fact that it had also reduced its H-1B workforce. 'Our H-1B applications are in no way related to the recent job eliminations in part because employees on H-1Bs also lost their roles,' a spokesman told me. He added that 78 percent of Microsoft's applications over the last 12 months were for 'existing employees.' Although these foreign workers are, in theory, intended to fill 'high-skill' roles, the data tell a different story. According to the Labor Department, 82 percent of Microsoft's H-1B applications for 2025 have been for positions the department classifies as Level I or II — entry or mid-level roles paid at or below the 34th wage percentile. To put that in simpler terms: Microsoft is paying 82 percent of its foreign workers less than the prevailing market rate for their positions. Even if Microsoft were able to find Americans willing to work for less than they're worth, Microsoft wouldn't be required to make any attempt to hire them — despite a widely held belief that H-1B employers are required to certify that they haven't been able to find Americans to employ. 'It is remarkable how many policy wonks, news organizations, and academics get this critical fact wrong,' Howard University Professor Ron Hira, an H-1B expert who has testified on the issue before Congress, told me in an interview. 'My experience suggests that more elites believe the falsehood than the truth. The program would look radically different and function much better if such a requirement existed.' It is little wonder that younger Americans suspect all of this labor importation may have something to do with the challenges they're facing. That applies not just in terms of employment, but also of problems ranging from the national housing shortage to the rising cost of car insurance to traffic congestion — which, as anecdotal evidence would have it, spontaneously fell amid immigration raids in Los Angeles last month. Naysayers will disagree, nitpick, or read legalese about how their foreign workers were onboarded years ago to rationalize the idea that Americans haven't been affected. And that's fine, but it isn't going to change the fact that voters aged 18-21 sided with Republicans by a double-digit margin in Yale's recent youth poll, largely due to these very concerns. Of course, those numbers could shift, particularly if Republicans fail to take action — likely pushing those young voters even further to the right. Aside from Vance, mainstream politicos have been largely silent on this issue — perhaps because they don't want to offend their corporate patrons. The best they can do is stay silent and hope that voters forget. The media appear to be fully on board with that plan. As of July, a search for stories involving the Office of Foreign Labor Certification published within the last year returned zero results from outlets including The New York Times, Washington Post, and NBC News, among many others. Contrast that with The Times' obsessive concern with the State Department terminating a little more than 1,300 employees. 'Cuts at State Department Demote Longtime U.S. Values,' the paper declared in the headline of a news (not opinion) article. It sounds like firing a federal worker is akin to an attack on the very fabric of America's values. By that measure, they must be the most American of us all. That must be nice, but it may not elicit much sympathy from those who have somehow been deemed less essential to the country than their foreign replacements.

Stoke Therapeutics and Biogen Announce First Patient Dosed in Phase 3 EMPEROR Study of Zorevunersen, a Potential Disease-Modifying Treatment for Dravet Syndrome
Stoke Therapeutics and Biogen Announce First Patient Dosed in Phase 3 EMPEROR Study of Zorevunersen, a Potential Disease-Modifying Treatment for Dravet Syndrome

Business Wire

time15 hours ago

  • Business Wire

Stoke Therapeutics and Biogen Announce First Patient Dosed in Phase 3 EMPEROR Study of Zorevunersen, a Potential Disease-Modifying Treatment for Dravet Syndrome

BEDFORD, Mass., & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine, and Biogen Inc. (Nasdaq: BIIB), today announced that the first patient has been dosed in the global Phase 3 EMPEROR study of zorevunersen for the treatment of Dravet syndrome. Zorevunersen, an investigational antisense oligonucleotide, has the potential to be the first disease-modifying treatment for Dravet syndrome. 'Our Phase 1/2 and open-label extension studies have provided a large dataset to support our understanding of zorevunersen and guide the EMPEROR study design, including dosing, duration and selection and powering of the endpoints,' said Barry Ticho, M.D., Ph.D., Chief Medical Officer of Stoke Therapeutics. 'Given the severity of this disease and the limitations of current treatments, the substantial and durable reductions in seizures and continuing improvements in cognition and behavior support our belief that zorevunersen may improve outcomes for patients with Dravet syndrome.' 'The initiation of the EMPEROR study is a critical milestone in zorevunersen's development,' said Katherine Dawson, M.D., Head of the Therapeutics Development Unit at Biogen. 'Despite treatment with available anti-seizure medicines, no approved medications currently address the underlying cognitive and behavioral aspects of this rare, genetic disease. Together with Stoke, we look forward to working in collaboration with the hope of bringing forward zorevunersen as the first disease-modifying treatment option, if approved, for Dravet syndrome.' EMPEROR Pivotal Phase 3 Design Summary Anticipated Enrollment: Patients with Dravet syndrome between the ages of 2 and up to 18 years of age with a confirmed variant in the SCN1A gene not associated with gain of function. Duration: Following an 8-week baseline period, participants will be randomized 1:1 to receive either zorevunersen or sham for a 52-week treatment period. Treatment Arms: Patients in the active treatment arm will receive two 70mg loading doses (Day 1 and Week 8) followed by two 45mg maintenance doses (Week 24 and Week 40). All patients will continue to receive standard of care medicines throughout the study. Primary Endpoint: Change in major motor seizure frequency measured at Week 28. Key Secondary Endpoints: Change in major motor seizure frequency measured at Week 52. Change in behavior and cognition as measured by the Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills measured at Week 52. Eligible participants will be offered ongoing treatment with zorevunersen as part of an open-label extension (OLE) study. 'Dravet syndrome is one of the most well studied genetic epilepsies so we know the significant and life-altering effects it can have on patients and their caregivers,' said Joseph Sullivan, M.D., FAES, principal investigator of the study and Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the University of California San Francisco. 'Providing additional relief from seizures remains an important clinical outcome, but the potential to address the underlying genetic cause to also address neurodevelopmental symptoms signals a fundamentally new way of treating the disease. The urgent need for treatments is evident in the high degree of interest in the EMPEROR study.' The EMPEROR clinical trial has initiated in the United States, United Kingdom, Japan and is planned for Europe. For more information on the EMPEROR study, please visit and About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Using Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels. Stoke's first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome and is currently being evaluated in a Phase 3 study. Stoke's initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency). Proof of concept has been demonstrated in other organs, tissues, and systems, supporting broad potential for the Company's proprietary approach. Stoke is headquartered in Bedford, Massachusetts. For more information, visit About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media - Facebook, LinkedIn, X, YouTube. About Dravet Syndrome Dravet syndrome is a severe developmental and epileptic encephalopathy (DEE) characterized by severe, recurrent seizures as well as significant cognitive and behavioral impairments. Most cases of Dravet are caused by mutations in one copy of the SCN1A gene, leading to insufficient levels of NaV1.1 protein in neuronal cells in the brain. More than 90 percent of patients continue to experience seizures despite treatment with the best available anti-seizure medicines. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. Developmental and cognitive impairments often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S. (~16,000), UK, EU-4 and Japan. 1 About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide that is designed to treat the underlying cause of Dravet syndrome by increasing NaV1.1 protein production in brain cells from the non-mutated (wild-type) copy of the SCN1A gene. This highly differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti-seizure medicines and to improve neurodevelopment, cognition, and behavior. Zorevunersen has demonstrated the potential for disease modification and has been granted orphan drug designation by the FDA and the EMA. The FDA has also granted zorevunersen rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation not associated with gain-of-function, in the SCN1A gene. Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under the collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico; Biogen receives exclusive rest of world commercialization rights. About the EMPEROR Study The EMPEROR Phase 3 Study (NCT06872125) is a global, double-blind, sham-controlled study evaluating the efficacy, safety and tolerability of zorevunersen in children ages 2 to <18 with Dravet syndrome with a confirmed variant in the SCN1A gene not associated with gain of function. The trial is expected to enroll participants across the United States, Japan, United Kingdom and European Union, with participants being randomized 1:1 to receive either zorevunersen via intrathecal administration or a sham comparator for a 52-week treatment period following an 8-week baseline period. Following the completion of the study, eligible participants will be offered ongoing treatment with zorevunersen as part of an OLE study. The primary endpoint of the study is percent change from baseline in major motor seizure frequency at week 28 in patients receiving zorevunersen as compared to sham. The key secondary endpoints are the durability of effect on major motor seizure frequency and improvements in behavior and cognition as measured by Vineland-3 subdomains, including expressive communication, receptive communication, interpersonal relationships, coping skills and personal skills. Additional endpoints include safety, Clinician Global Impression of Change (CGI-C), Caregiver Global Impression of Change (CaGI-C), and the Bayley Scales of Infant Development (BSID-IV). For more information, visit Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the 'safe harbor' provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the ability of zorevunersen to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; the design, timing and results of the Phase 3 EMPEROR study; and the expectations and potential benefits of Stoke's collaboration with Biogen. Statements including words such as 'anticipate,' 'could,' 'expect,' 'plan,' 'will,' or 'may' and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause Stoke's results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: Stoke's ability to advance, obtain regulatory approval and ultimately commercialize its product candidates; that if Stoke's collaborators were to breach or terminate their agreements, it would not obtain the anticipated financial or other benefits; the possibility that Stoke and Biogen may not be successful in their development of zorevunersen and that, even if successful, they may be unable to successfully commercialize zorevunersen; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; Stoke's ability to protect its intellectual property; Stoke's ability to fund development activities and achieve development goals through mid-2028; and the other risks and uncertainties described under the heading 'Risk Factors' in Stoke's Annual Report on Form 10-K for the year ended December 31, 2024, its quarterly reports on Form 10-Q, and the other documents it files with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and Stoke undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Biogen Safe Harbor This news release contains forward-looking statements, including relating to the potential, benefits, safety and efficacy of Zorevunersen; the potential of Biogen's commercial business and pipeline programs, including Zorevunersen; the anticipated benefits and potential of Biogen's collaboration arrangement with Stoke Therapeutics; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by such words as 'aim,' 'anticipate,' 'assume,' 'believe,' 'contemplate,' 'continue,' 'could,' 'estimate,' 'expect,' 'forecast,' 'goal,' 'guidance,' 'hope,' 'intend,' 'may,' 'objective,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'prospect,' 'should,' 'target,' 'will,' 'would,' and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. These forward-looking statements are based on management's current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward-looking statements will be realized in whole or in part. We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document, including, among others, uncertainty of long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans and prospects relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; our ability to effectively implement our corporate strategy; the successful execution of our strategic and growth initiatives, including acquisitions; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; and any other risks and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned 'Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise. Reference: Based on Stoke Therapeutics' preliminary estimates, which scaled annual incidence to prevalence using country-specific live birth rates over the past 85 years and adjusted for Dravet-specific mortality. The estimate is based on incidence rates published by .

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